The FDA and Multiple Sclerosis

Sadly, there is more blood on the hands of the FDA. Its as if these people care only about their regulatory program rather than the lives and health of people. Of course, that couldn’t be – that would be unspeakable.

The Wall Street Journal has a piece on a drug that is capable of dramatically helping some people with multiple sclerosis. The drug has been approved in “30 countries, including Canada, Australia and all members of the European Union. But on Dec. 27, Food and Drug Administration reviewers at the division level (subject to a final decision by top officials) rejected an application to use the drug here to combat MS.” Sadly, the division level reviewers are rarely if ever overruled.

Why did the FDA lords refuse to approve the drug?

The primary reason FDA reviewers gave for rejecting Lemtrada was that the studies demonstrating the drug’s efficacy did not conform to the agency’s standard requirement of double-blind, placebo-controlled drug trials—where some patients, unbeknownst to themselves and their doctors, receive placebo treatments. There are excellent reasons for the standard approach, but only up to a point. Lemtrada and many established MS treatments have immediate side effects, such as nausea and headaches, that are well known to doctors and patients. A double-blind trial would not really be blind. Patients on a placebo would promptly discover that they were the “controls,” and many would decline to participate further—scrambling the statistical comparison with patients receiving real treatments.

The article continues:

For Lemtrada, the FDA reviewers announced that the trials were not “adequate and well-controlled.” They are now demanding another round of trials, with somewhat different procedures, that would take years and cost at least $100 million. Given the magnitude of the results of the already completed trials, the additional trials could add nothing to answering the regulatory question of whether Lemtrada is suitable for clinical use against MS.

If one of my family member needed this drug, I would be protesting. I would find out who the peope were who turned it down and I would protest in front of their house with dramatic images of the infirmity this disease causes. Let these doctors explain their actions to the children and their neighbors.

The Republican Party is supposed to be the party that resists regulation but alas they are pretty lame. There ought to be bills being introduced in Congress to address these problems. Here are a couple of places to start:

1. Off label uses – where the drug is approved for treatment A, but not treatment B – should not only be allowed but should be permitted to be advertised. The drug is approved as safe and the medical industry is perfectly able to determine whether it is effective for other uses. The FDA goes after advertising of these off label uses with a vengence, denying truthful information to people.

2. Drugs that are approved in a certain percentage of a group of other countries, such as in Canada and Europe, should be presumptively approved. The FDA should only then be able to disapprove the drug if it can show that there is a problem.

These reforms are to my mind no brainers, but they will be resisted by the FDA and others tooth and nail. It is time to start the battle.

And I know the perfect person to lead the charge. There is a libertarian Senator who is a doctor who has a talent for publicizing important matters. This should be Rand Paul’s cause.

Reader Discussion

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on February 04, 2014 at 11:07:43 am

While I do not wish to detract from Professor Rappaort's main point, the case of Lemtrada may not be the best example. The drug, a monoclonal antibody, had been approved for use in the United States for treatment of certain types of leukemia and lymphoma. Physicians could prescribe it off label for treatment of multiple sclerosis. The manufacturer, Sanofi, withdrew the agent from the U.S. market to prevent this off label use and to allow it to be rebranded and repriced as a treatment specifically for multiple sclerosis. In the quaint language of a business story on the move, the manufacturer withdrew the cancer treatment "from markets on both sides of the Atlantic in order to optimise the pending launch of the drug as a treatment for multiple sclerosis[.]"

MS patients may be getting screwed, but there is more to it than just bureaucratic foibles.

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on February 04, 2014 at 11:27:59 am

I don't know enough about the decision to withdraw and rebrand to comment. But the restrictions on off label use -- including not allowing companies to advertise and even the unwillingness of some companies even to answer questions asked about the off label use -- make its use in many cases decidedly inferior.

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Mike Rappaport
on February 04, 2014 at 11:35:19 am

Having read the linked article, I do agree that more is going on than just bureaucratic foibles. But I also think that bureaucratic foibles are still part of it.

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Mike Rappaport
on February 04, 2014 at 14:18:01 pm

The regulation of food and drugs provides a quintessential opportunity for exploring the relationship between bureaucracy and liberty. In theory, it is the prototype of regulating for the public welfare, protecting the masses from threats beyond the expertise of the common man to evaluate and manage for himself.

The FDA regulates in the legacy of thalidomide, the mutagen that led to limbless babies in Europe, but was kept from our shores by bureaucratic vigilance. That was good, but the agency also deprives other patients of drugs that have either proven benefit to them, or potential benefit, based on nebulous population risks. Examples of such drugs include cisapride, cerivastatin, and rofecoxib (Vioxx). Some patients who experienced demonstrated benefits from these agents were subsequently denied them because of the presumed risks to others. The fundamental philosophical problem for the libertarian is who should determine the risk/benefit relationship for a particular therapy in a particular patient? Should "experts" and bureaucratic chair-warmers be allowed to deprive a patient of a medicine that may potentially provide a cure, or which makes the burden of disease more bearable because they have decided that the risk to the average, standardized person is not worth it? Should people have the liberty to chance a bad outcome when the alternatives have been tried and failed? Do "black box warnings" and caveat emptor suffice?

The pro-regulatory arguments are numerous: that the everage person, or even average physician lacks the background to make fully informed decisions; that in the absence of firm oversight, drug companies will advertise and prey on desperate and unsophisticated patients, returning to the patent medicine scams of bygone days. Adverse drug effects could yield epidemics of undesirable otcomes, like the birth defects associated with thalidomide, that then become more generalized societal burdens.

On the other hand, "experts" are not always so. Saccharine was thought to be a carcinogen in humans because it was found to be so in rodents. The extrapolation proved to be wrong. Hormone replacement therapy was thought to stave off any number of senescent maladies, with little down-side. Oops. Furthermore, the libertarian may ask why the government should have any more say in how he copes with burden of disease than in determining who he should marry. Heroin is available by prescription in England; the risks and benefits apparently left to the therapeutic relationship between patient and provider for resolution. Bureaucracies are prone to hidden agendas. It is not hard to imagine that the FDA did not like Sanofi's manipulative business tactic, and thought to its chain a little.

I am not advocating a position here. The FDA does some good and some bad. Some people are better off because of it and for others the opposite is true, but for anyone who is concerned about the relationship between the citizen and the state the regulation of health and health services provides a lot to contemplate.

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on February 05, 2014 at 13:22:05 pm

The FDA regulates in the legacy of thalidomide, the mutagen that led to limbless babies in Europe, but was kept from our shores by bureaucratic vigilance.

Correction: kept from our shores by bureaucratic lassitude.

Bureaucratic vigilance would have resulted in thalidomide's swift disapproval by the FDA. Instead, the application for thalidomide was left sitting on a shelf as it was processed with all sub-deliberate speed for which the Drug DMV is famous. Only some time after the disaster had been widely reported in the popular press did the Drug DMV act. That is not vigilance, even of the slow-walking bureaucratic sort.

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Micha Elyi
on February 07, 2014 at 10:30:27 am

I read the comments regarding the FDA's refusal to approve Lemtrada with interest.
As a physician who only treats MS patients, I feel that that the FDA has done a great disservice to my patients with their decision. It was not based on the efficacy of the drug or it's potential side-effects but rather on the study methodology . However, realistically the study could not have done any other way. In my mind to deprive patients of this drug is shameful. I know the history of this monoclonal antibody and from long prior experience, although approved for other uses, it will not be available "off-label" because insurance companies will refuse to pay for it. I have many patients who have failed the other disease- modifying drugs and were waiting for Lemtrada. What are they to do?

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Amos Katz

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